Ionis Eplontersen gets USFDA orphan drug designation

Eplontersen is a LIgand-Conjugated Antisense (LICA) investigational medicine currently in Phase 3 clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). In December 2021, Ionis announced a strategic collaboration with AstraZeneca to develop and commercialize eplontersen.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis. It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms. Hereditary ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.

"Receiving FDA orphan drug status for eplontersen underscores the significant unmet need for novel treatment options for people living with transthyretin-mediated amyloidosis. We look forward to working closely with regulators, clinical investigators, patients and their families to advance this important medicine and make it available to those who may benefit from it," said Richard S. Geary, Ph.D., executive vice president and chief development officer at Ionis.